The patient's own bone marrow-derived stromal cells: disease modifiers in (neuro)degenerative disorders

Research output: ThesisDoctoral ThesisExternal prepared

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Abstract

The aim of this thesis was to identify a standardized stem cell product that can be used to treat secondary inflammation in neurodegenerative diseases to improve the clinical outcome. In a comprehensive literature search, the concept of the patient’s own fresh naïve stem cells was developed, tested in animal models in order to prepare for the first clinical trials in patients suffering a neurodegenerative disorder. A standardized human stem cell product (Neuro-Cells®) was developed and patented, which is produced on the Brightlands campus in the good manufacturing product (GMP) facility. In animal experiments, using human GMP processed stem cells (Neuro-Cells), the researchers were able to proof safety and efficacy in the indications spinal cord injury, amyotrophic Lateral sclerosis and Frontotemporal lobar degeneration. The stem cells act as an anti-inflammatory drug and proved to be superior to methylprednison, Celecoxib and Riluzole. They concluded that in preclinical trials, a transplantation with human stem cells might be seen as a disease modifier, acting on the secondary inflammatory mechanisms and thereby decreasing disability. With the results of the spinal cord injury experiments, they were able to start a phase I study with chronic spinal cord injured patients together with the Hospital Nacional de Parapléjicos de Toledo in Spain. All 10 patients are treated with their stem cells and so far no side effects were reported. A multi-centre Phase II/III is planned to start in June 2021.
Original languageEnglish
Awarding Institution
  • Maastricht University
Supervisors/Advisors
  • Kramer, Boris, Supervisor
  • Wolters, Erik Ch, Supervisor, External person
  • Strekalova, Tatiana, Co-Supervisor
  • Mey, Jörg, Co-Supervisor
Award date9 Apr 2021
Place of PublicationMaastricht
Publisher
Print ISBNs9789090344706
DOIs
Publication statusPublished - 2021

Keywords

  • autologous stem cells
  • neurodegeneration
  • spinal cord injury
  • naïve stem cells
  • secondary inflammation
  • Amyotrophic Lateral Sclerosis
  • orphan drug status

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